WORLDSymposium 2022 New Treatment Award

In recognition of achieving major milestones, WORLDSymposium marks important progress with two New Treatment Awards in 2022. This award honors “new treatments” that are viewed as providing value to patients with lysosomal diseases, with general acceptance as evidenced by approval of the U.S. Food and Drug Administration, European Medicines Agency, and other regulatory authorities.

This year WORLDSymposium recognizes two important achievements in therapy for lysosomal diseases for attaining regulatory approval. The WORLDSymposium 2022 New Treatment Award will be presented to Sanofi Genzyme for avalglucosidase alfa-ngpt (Nexviazyme®) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA), and to JCR Pharmaceuticals for pabinafusp alfa (IZCARGO®), which provided clinical data meriting approval by the Ministry of Health, Labour and Welfare (MHLW) in Japan.

Nominations for this award will be accepted up until the meeting, and may be submitted for any therapy achieving regulatory approval. Eligible and verified new treatments will be recognized with the WORLDSymposium 2022 New Treatment Award presented on Thursday, February 10, 2022, at 7:30 AM followed by a full day of Contemporary Forum abstract presentations.

Past Award Recipients:

2018 cerliponase alfa (Brineura™, BioMarin)
2018 vestronidase alfa (MEPSEVII™, Ultragenyx)
2017 migalastat (Galafold™, Amicus)
2016 sebelipase alfa (Kanuma™, Alexion)
2015 eliglustat (Cerdelga™, Sanofi Genzyme)
2015 idursulfase beta (Hunterase®, GC Pharma)
2015 cysteamine bitartrate (PROCYSBI™, Horizon Therapeutics)
2015 elosulfase alfa (Vimizim®,  BioMarin)